Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Deflazacort · 6 trials · 4 indications
Pharmacokinetic parameters such as the maximum observed plasma concentration (Cmax), time to Cmax (Tmax), the area under the plasma concentration versus time curve from time 0 (predose) to the last quantifiable time point (AUClast), AUC from time 0 (predose) to time infinity (AUCinf), the elimination rate constant (λz), and terminal elimination half-life (t½) will be calculated
The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active
Long-term safety and tolerability will be characterized by the number, frequency, and severity of adverse events from Day 1 through End of Study or Early Termination.
| Arm | Type | Description |
|---|---|---|
| Deflazacort, fasted | EXPERIMENTAL | 36mg of Deflazacort with 240 ml of room-temperature, non-carbonated water in Fasted state. |
| Deflazacort, high-fat meal | EXPERIMENTAL | 36mg of Deflazacort with 240 ml of room-temperature, non-carbonated water with high fat meal served 30 minutes prior to dosing. |
| Deflazacort, crushed, fasted | EXPERIMENTAL | 36mg of Deflazacort crushed and mixed with apple sauce. |
| Deflazacort alternate strength,fasted | EXPERIMENTAL | Investigational Formulation Deflazacort tablet (6 X 6mg). |
| Deflazacort suspension with apple juice | EXPERIMENTAL | Deflazacort oral suspension (36mg) mixed with 100ml apple juice in fasted state. |
| Deflazacort | EXPERIMENTAL | This is an open label and single period study , dosed with 0.9mg/kg Deflazacort. |
| Hepatic Impairment | EXPERIMENTAL | Eight (8) subjects with moderate hepatic insufficiency (a score of 7 to 9, on the Child-Pugh scale) will receive one 18 mg dose of deflazacort |
| Healthy Volunteer | EXPERIMENTAL | Eight (8) healthy subjects. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the moderate hepatic impaired cohort; will receive 18 mg dose of deflazacort |
| Renal Impairment | EXPERIMENTAL | Eight (8) subjects with ESRD on HD will receive one 18 mg dose of deflazacort. |
| Healthy Volunteers | EXPERIMENTAL | Eight (8) healthy subjects with estimated creatinine clearance (CLcr) ≥ 90 mL/min. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the ESRD cohort. Subjects will receive one 18 mg dose of deflazacort. |
| Cohort A Deflazacort and Rifampin | EXPERIMENTAL | Subjects will recieve one 18 mg dose of deflazacort on Day 1, Period 1 and Day 10, Period 2; cohort A. Subjects will receive once daily dosing of rifampin on Day 1, Period 2 through Day 10, Period 2. |
| Cohort B Deflazacort and Clarithromycin | EXPERIMENTAL | Subjects will recieve one 18 mg dose of deflazacort on Day 1, Period 1 and Day 4, period 2; cohort B. Subjects will receive twice daily dosing of clarithromycin on Day 1, Period 2 through Day 4, Period 2. |
| Name | Type | Description |
|---|---|---|
| Deflazacort | DRUG | - |
| Deflazacort and rifampin | DRUG | Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized completely and rapidly to the active drug 21-desacetyldeflazacort (21 desacetyl-DFZ). The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone. Rifampin is a potent inducer of drug metabolism by inducing a variety of hepatic and intestinal CYP enzymes, especially CYP3A4. Rifampin is a semi-synthetic antibiotic. |
| Deflazacort and Clarithromycin | DRUG | Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects. It is an inactive pro-drug which is metabolized completely and rapidly to the active drug 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone. Clarithromycin is a semi-synthetic macrolide antibiotic. Clarithromycin is active in vitro against a variety of aerobic and anaerobic gram-positive and gram-negative bacteria as well as most mycobacterium avium complex (MAC) bacteria. Additionally, the 14-OH clarithromycin metabolite also has clinically significant antimicrobial activity. Clarithromycin is indicated for the treatment of mild to moderate infections such as pharyngitis/tonsillitis. |
Inclusion Criteria: * Healthy males and females ≥ 18 and ≤ 55 at the time of screening. * Body mass index (BMI) ≥ 18.0 and ≤ 32.0 kg/m2, inclusive, at screening and Day -1. * Women of nonchildbearing potential, defined as tubal ligation, hysterectomy, postmenopausal (amenorrhea for \> 1 year; confi...
Deflazacort is a small molecule being developed by PTC Therapeutics for use in Duchenne Muscular Dystrophy, as well as in studies involving healthy volunteers and patients with renal or hepatic impairment. It is currently in Phase 1 clinical development.
Deflazacort is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol PTCT. The company is conducting Phase 1 clinical trials to evaluate the drug's pharmacokinetics in various patient populations.
Deflazacort is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing trials are focused on evaluating its pharmacokinetics and effects in healthy volunteers and patients with hepatic or renal impairment.
Deflazacort has been studied in four completed Phase 1 clinical trials: NCT02286609 (hepatic impairment), NCT02286622 (renal impairment), NCT02286635 (drug interactions with rifampin and clarithromycin), and NCT02485431 (food effect and bioavailability). These trials enrolled a total of 103 participants.
Deflazacort is also known by the brand name Emflaza. It is a corticosteroid used to treat Duchenne Muscular Dystrophy. The drug is being studied in Phase 1 trials to understand its pharmacokinetics in different populations, including those with organ impairment.